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Muscle & Nerve|April 9, 2024
Debamestrocel multimodal effects on biomarker pathways in amyotrophic lateral sclerosis are linked to clinical outcomesStacy R Lindborg, Namita A Goyal, Jonathan Katz, et al.
Neurology|October 4, 2019
Prospective natural history study of C9orf72 ALS clinical characteristics and biomarkersAlexander J Cammack, Nazem Atassi, Theodore Hyman, et al.
Proceedings of the National Academy of Sciences of the United States of America|October 31, 2013
Targeted degradation of sense and antisense C9orf72 RNA foci as therapy for ALS and frontotemporal degenerationClotilde Lagier-Tourenne, Michael Baughn, Frank Rigo, et al.
Frontiers in Cellular Neuroscience|June 22, 2023
Moderate intrinsic phenotypic alterations in C9orf72 ALS/FTD iPSC-microglia despite the presence of C9orf72 pathological featuresIleana Lorenzini, Eric Alsop, Jennifer Levy, et al.
Nature Medicine|September 5, 2022
Transplantation of human neural progenitor cells secreting GDNF into the spinal cord of patients with ALS: a phase 1/2a trialRobert H Baloh, J Patrick Johnson, Pablo Avalos, et al.
Neurology|January 23, 2015
Novel mutations expand the clinical spectrum of DYNC1H1-associated spinal muscular atrophyMariacristina Scoto, Alexander M Rossor, Matthew B Harms, et al.
Nature Neuroscience|April 2, 2014
Mutations in the Matrin 3 gene cause familial amyotrophic lateral sclerosisJanel O Johnson, Erik P Pioro, Ashley Boehringer, et al.
JAMA Neurology|November 23, 2020
Effect of Ezogabine on Cortical and Spinal Motor Neuron Excitability in Amyotrophic Lateral Sclerosis: A Randomized Clinical TrialBrian J Wainger, Eric A Macklin, Steve Vucic, et al.
Science (New York, N.Y.)|February 21, 2015
Exome sequencing in amyotrophic lateral sclerosis identifies risk genes and pathwaysElizabeth T Cirulli, Brittany N Lasseigne, Slavé Petrovski, et al.
Nature Genetics|December 7, 2021
Common and rare variant association analyses in amyotrophic lateral sclerosis identify 15 risk loci with distinct genetic architectures and neuron-specific biologyWouter van Rheenen, Rick A A van der Spek, Mark K Bakker, et al.
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